FDA Grants Accelerated Approval to Futibatinib for Locally Advanced, Metastatic Cholangiocarcinoma


Futibatinib (Lytgobi, Taiho Oncology, Inc) is indicated for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements.

The FDA has granted accelerated approval to futibatinib (Lytgobi, Taiho Oncology, Inc) for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements.1

Futibatinib is an investigational, oral, potent, selective, and irreversible tyrosine kinase inhibitor of FGFR1, 2, 3 and 4. It was previously granted FDA priority review as a treatment for patients with advanced solid tumors with FGFR1-4 genetic aberrations, including cholangiocarcinoma, previously treated with chemotherapy or other therapies.

Futibatinib selectively and irreversibly binds to the ATP binding pocket of FGFR1-4, which inhibits FGFR-mediated signal transduction pathways, decreases tumor cell proliferation, and increases tumor cell death in tumors with FGFR1-4 genetic aberrations, according to Taiho Oncology.

"Lytgobi is an effective, well-tolerated therapy for patients with intrahepatic cholangiocarcinoma that can be taken orally," said Tim Whitten, president and chief executive officer of Taiho Oncology, Inc, in a press release."This approval is an important milestone for patients and may provide hope for improved outcomes. As someone whose family has been impacted by cholangiocarcinoma, I'm acutely aware of the impact this disease can have on the patient and their loved ones."2

The approval of futibatinib was based on data from the multicenter, open-label, single-arm, phase 2 FOENIX*-CCA2 trial (TAS-120-101; NCT02052778) in 103 patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma harboring a FGFR2 gene fusion or other rearrangement. Presence of FGFR2 fusions or other rearrangements was determined via next generation sequencing testing. Patients were administered 20 mg of futibatinib orally once daily until disease progression or unacceptable toxicity.

The results of the trial showed that futibatinib produced an overall response rate of 42% (95% CI, 32%-52%) by independent central review and RECIST v1.1 criteria, with all responders experiencing partial responses. Median duration of response (DOR) with futibatinib was 9.7 months (95% CI, 7.6-17.1). Further, 72% of patients experienced a DOR that lasted for at least 6 months, and 14% had a response that lasted for at least 12 months. The median time to response was 2.5 months (range, 0.7-7.4).

The FDA granted Breakthrough Therapy Designation to futibatinib for patients with previously treated locally advanced or metastatic cholangiocarcinoma in 2021.

The most common adverse events reported in 20% or more of patients were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythrodysesthesia syndrome, and vomiting.

"Lytgobi is a key example of the potential of precision medicine in iCCA and represents another advance in the treatment of this rare and challenging disease," said lead trial investigator Lipika Goyal, MD, MPhil, medical oncologist, of the Massachusetts General Hospital Cancer Center, in a press release. "I am encouraged that treatment options continue to expand and evolve for this disease through the dedicated efforts of many over several years."2


1. FDA grants accelerated approval to futibatinib for cholangiocarcinoma. FDA. News release. September 30, 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-futibatinib-cholangiocarcinoma?utm_medium=email&utm_source=govdelivery

2. FDA Approves Taiho's LYTGOBI® (futibatinib) Tablets for Previously Treated, Unresectable, Locally Advanced or Metastatic Intrahepatic Cholangiocarcinoma. Taiho Oncology, Inc. News release. September 30, 2022. https://www.prnewswire.com/news-releases/fda-approves-taihos-lytgobi-futibatinib-tablets-for-previously-treated-unresectable-locally-advanced-or-metastatic-intrahepatic-cholangiocarcinoma-301638254.html

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